Application US20200165630

COMPOSITIONS FOR THE TREATMENT OF DISEASE
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US20200165630

1. An AAV particle comprising a capsid and a viral genome, said viral genome comprising at least one inverted terminal repeat (ITR) region and a payload region, said payload region comprising a regulatory sequence operably linked to at least a first nucleic acid segment, said first nucleic acid segment encoding one or more polypeptides selected from the group consisting of any member given in Tables 3-42 and fragments thereof. (19) (0)

2. The AAV particle of claim 1, wherein the capsid is selected from the group of serotypes consisting of Table 1. (1) (0)

5. The AAV particle of claim 1, wherein the viral genome is single stranded. (0)

6. The AAV particle of claim 1, wherein the viral genome is self-complementary. (0)

7. The AAV particle of claim 1, wherein at least one region of the viral genome is codon-optimized. (1) (0)

20. The AAV particle of claim 1, wherein the first nucleic acid segment encodes an antibody, having at least 95% identity to any of the sequences selected from the group consisting of SEQ ID NO: 2948-9220 (Tables 3-42). (0)

49. The AAV particle of claim 1, wherein the viral genome comprises 2 ITR regions. (0)

50. The AAV particle of claim 1, wherein the at least one ITR region is derived from the same parental serotype as the capsid. (0)

51. The AAV particle of claim 1, wherein the at least one ITR region is derived from a different serotype as the capsid. (0)

52. The AAV particle of claim 1, wherein the at least one ITR region is derived from AAV2. (0)

53. The AAV particle of claim 1, wherein the at least one ITR region is 100-150 nucleotides in length. (0)

54. The AAV particle of claim 1, wherein the at least one ITR region is 102 nucleotides in length. (0)

55. The AAV particle of claim 1, wherein the at least one ITR region is 140-142 nucleotides in length. (0)

56. The AAV particle of claim 1, wherein the at least one ITR region is 140 nucleotides in length. (0)

57. The AAV particle of claim 1, wherein the at least one ITR region is 141 nucleotides in length. (0)

58. The AAV particle of claim 1, wherein the at least one ITR region is 142 nucleotides in length. (0)

59. The AAV particle of claim 1, wherein the viral genome further comprises an intron or stuffer sequence. (0)

60. A method of producing an antibody in a subject comprising administering the AAV particle of claim 1 to said subject, with the proviso that the antibody is not a virus neutralizing antibody. (0)

61. A method of producing an antibody in a subject comprising administering the AAV particle of claim 1 to said subject, with the proviso that the antibody is not an HIV or HCV virus neutralizing antibody. (0)

62. The AAV particle of claim 1, wherein the payload region of the viral genome comprises a second nucleic acid segment, said second nucleic acid segment encoding an aptamer, siRNA, saRNA, ribozyme, microRNA, mRNA or combination thereof. (3) (0)

21. An AAV particle comprising a capsid and a viral genome, said viral genome comprising at least one inverted terminal repeat (ITR) region and a payload region comprising a regulatory sequence operably linked to at least a first nucleic acid segment, said first nucleic acid segment encoding a bispecific antibody derived from any of the sequences listed in Tables 3-42 or portions or fragments thereof. (1) (0)

27. A pharmaceutical composition comprising an AAV particle of any of the preceding claims in a pharmaceutically acceptable excipient. (2) (0)

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Application US20200165630

COMPOSITIONS FOR THE TREATMENT OF DISEASE

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